GENE THERAPY APPROVED! SMA A CHANGED DISEASE!May 25, 2019 by john
Gene Therapy FDA APPROVED! And, because of your support, the Gwendolyn Strong Foundation helped do this!
Today, AveXis, a Novartis company, announced the FDA has officially approved Zolgensma® for children under two. Today, babies newly diagnosed with the #1 genetic killer of young children now have a FUTURE! SMA is forever CHANGED. Ten years ago, with little more than hope, we rallied supporters around the globe to help make this day a reality. Families ran races, held bowling nights and spaghetti feeds, a few even shaved their heads... all to raise much-needed funding to see this revolutionary research pursued.
Children born with Type 1 SMA (like Gwendolyn) are now able to walk, talk, speak, breathe... "Something like this has never been achieved before." Dr. Jerry Mendell, principal investigator in the Center for Gene Therapy at The Research Institute of Nationwide Children's. Zolgensma is designed to address the genetic root cause of SMA by replacing the defective or missing SMN1 gene to halt disease progression with a single, one-time infusion. This one-time infusion introduces a virus to act as a “vector’’ and transfer a permanent replacement of the defective gene into the body. Early diagnosis and treatment are necessary for the greatest impact. A separate trial is underway for older children already affected by the disease.
This news is groundbreaking on so many levels. And gene therapy for SMA is just the first of many genetic diseases to benefit. A new door is officially opened in science and it is mind-blowing. We are so grateful for the incredible scientists, the nonprofit, hospital, and university partners, AveXis and Novartis, the NIH and FDA, and, of course, the brave families who took the leap and enrolled in the clinical trial.
This was one of the most material grants the Gwendolyn Strong Foundation awarded and to see it come to fruition is incredible! THANK YOU to everyone who has been part of this. To our partners at FightSMA who believed in gene therapy long before it had proven itself. To all who supported the Gwendolyn Strong Foundation, worked hard and sacrificed to change the future. And, to all the children whose lives inspired action, your legacy has made the future brighter. NEVER GIVE UP.